Clinical characteristics and outcomes of adult alveolar rhabdomyosarcoma (ARMS) patients on front-line therapies: An MD Anderson Cancer Center (MDACC) case series.
Authors: Michael Nakazawa, J Andrew Andrew Livingston, Andrew Justin Bishop, Maria Alejandra Zarzour, Neeta Somaiah, Ravin Ratan, Jonathan Benjamin Gill, Elizabeth Urquiola, Kristi Posey, Beverly Ashleigh Guadagnolo, Richard Greg Gorlick, Robert S. Benjamin, Shreyaskumar Patel, Anthony Paul Conley
Journal: Journal of Clinical Oncology (2022)

e23548 Background: Rhabdomyosarcomas are the most common soft tissue sarcoma in children, and the prognosis of pediatric ARMS has improved with the use of multi-modality therapies. However, ARMS in adults is rare, and long-term outcomes continue to be poor. This study aimed to evaluate clinical outcomes of an adult ARMS population on different front-line systemic chemotherapies, particularly the vincristine/doxorubicin/ifosfamide (VDI) regimen. Methods: Adult fusion-positive confirmed ARMS patients over the age of 18 years (y) treated at MDACC from 2004 to 2018 were identified in our patient registry. Descriptive clinical statistics including staging, front-line chemotherapy, multimodal therapy usage, and survival analyses were performed. Results: 49 patients were identified, with mean age of 34.9 y (range 18y - 67y), and 53% were male. Most patients were white (53%, 26 pts), and the most common primary tumor site was the parameningeal space (63%; 31 pts). Patients were either intermediate (67%) or high clinical risk (33%). Most patients were IRSG clinical group IIIa (36%), IIIb (20%) or IV (33%) and were classified clinical stage 3 (49%) or 4 (33%). Of all patients at diagnosis, 71% had nodal disease and 32% were metastatic. Radiotherapy and surgery were given with upfront chemotherapy in 33 pts (67%) and 24 pts (49%) respectively, with 19 patients receiving both. Median OS for the entire cohort was 3.6 years. Doxorubicin containing chemotherapy regimens trended to worse OS than non-doxorubicin containing regimens (2.3 yrs vs 4.0 yrs, p = 0.355). Comparing patients who received VDI (19 pts) vs non-VDI (30 pts; 13 received Actinomycin D, 12 received doxorubicin in different regimens, and 5 received neither), median OS was 1.8 yrs vs 3.8 yrs (p = 0.283) respectively. There were similar number of front-line chemotherapy cycles (8.5 vs 9.5 cycles), high clinical risk (26% vs 37%) and metastatic disease (21% vs 36%) in the VDI vs non-VDI cohorts. Patients receiving upfront radiation had improved survival (3.7 vs 1.5 yr, p = 0.01), but this is likely confounded by those with metastases being less likely to receive upfront radiation. Conclusions: In this single center retrospective analysis of adult ARMS patients, survival outcomes continue to be similar to historical outcomes. There was no statistically significant OS difference in patients who did or did not receive doxorubicin containing front-line chemotherapy regimens, or in particular VDI therapy, although there was a trend to decreased OS. However, limitations to this study include limited sample size, non-randomization to treatment selection, and possible biases in patient selection for different chemo regimens. Based on these observations, randomized prospective studies are necessary to delineate which frontline chemotherapy regimen is most beneficial in this rare tumor in adults.